What’s Next in Obstructive Hypertrophic Cardiomyopathy Treatment?

Treatment options for obstructive hypertrophic cardiomyopathy (oHCM) are shifting from general symptom management toward therapies that target the biological mechanisms of the disease. While there is currently no cure for oHCM, a combination of newly approved medications, emerging drug classes, and less-invasive surgical techniques is expanding the options available to patients.

The most significant recent advancement is the development of cardiac myosin inhibitors. These medications target the underlying cause of the condition by slowing the interaction between the proteins actin and myosin, which are essential for muscle function. This mechanism prevents the heart muscle from contracting too forcefully, resulting in a more normal contraction pattern.

Mavacamten (Camzyos) became the first medication to target the root cause of oHCM upon its approval in 2022. In December 2025, the U.S. Food and Drug Administration (FDA) approved a second inhibitor in this class, aficamten (Myqorzo).

Both mavacamten and aficamten have demonstrated similar efficacy in reducing common symptoms such as chest pain and shortness of breath. A third inhibitor, delocamten, is currently in phase 2 clinical trials and is designed to offer a more flexible dosing schedule than the first two medications.

Emerging Pharmacological Therapies

Beyond myosin inhibitors, researchers are investigating other drug classes to improve heart function. Sarcomere modulators represent a different approach by focusing on the heart’s ability to fill with blood rather than how it contracts.

The investigational sarcomere modulator EDG-7500 is currently in a phase 2 clinical trial. Early results suggest the drug is safe and improves the relaxation phase of the heart muscle. If phase 3 trials are successful, experts anticipate potential FDA approval around 2030.

sodium-glucose cotransporter 2 (SGLT2) inhibitors are being studied for their potential utility in oHCM. These drugs, which include sotagliflozin, typically work via metabolic pathways to remove excess sugar from the body and have proven effective in treating various types of heart failure.

Dr. Anjali Tiku Owens, the medical director of the Center for Inherited Cardiac Disease at Penn Medicine in Philadelphia, noted that these drugs appear to have beneficial effects on cardiac function, although the exact mechanism for oHCM is not yet fully understood.

A phase 3 clinical trial for sotagliflozin is scheduled to conclude in August 2026. If the results are positive, the drug could be submitted for FDA approval for oHCM shortly after the trial’s completion.

Advancements in Surgical Intervention

The prevalence of invasive surgeries for oHCM has decreased since the introduction of cardiac myosin inhibitors. However, surgery remains necessary for some patients, leading to the development of procedures that reduce risk and recovery time.

Traditional septal myectomy is an open-heart surgery that removes a portion of the thickened heart muscle and requires the breastbone to be opened. A less-invasive alternative, transapical beating heart septal myectomy, uses a smaller incision and does not require the breastbone to be cracked.

According to Dr. Sara Saberi, an assistant professor in the division of cardiovascular medicine at the University of Michigan in Ann Arbor, this approach is a game changer. While it has been shown to be safe and effective in clinical trials in China, it is still awaiting potential approval in the United States.

Another emerging option is minimally invasive electro septal myectomy. Small studies indicate this method is safe and effective, utilizing a tool similar to an apple corer to precisely remove specific amounts of heart muscle.

Long-Term Research and Gene Therapy

While current treatments focus on management, gene therapy offers the long-term possibility of addressing the genetic origins of oHCM. Scientists have identified nearly 30 genes involved in the development of the disease, most of which affect heart muscle contraction.

Future applications of gene editing technologies, such as CRISPR, could potentially silence faulty genes or replace them with functional ones. This would allow the heart muscle to contract normally from a genetic level.

Despite the potential, gene therapy is in its earliest stages. Dr. Owens stated that the field is at the very beginning, with only the first few patients participating in phase 1 clinical trials. Significant research will be required before gene therapy becomes an approved clinical treatment.

Accessing New Treatments

For patients whose symptoms are not sufficiently controlled by approved therapies like mavacamten or aficamten, clinical trials provide a pathway to access cutting-edge treatments.

Patients can identify ongoing research opportunities through the following steps:

• Searching for active oHCM studies at ClinicalTrials.gov.
• Consulting with a cardiologist to determine eligibility for specific trials.
• Discussing the potential risks and benefits of investigational drugs like delocamten or EDG-7500.

The current medical landscape for oHCM is characterized by a move toward precision medicine. By combining targeted pharmacological inhibitors with refined surgical techniques, clinicians are now able to offer more personalized care to patients with limiting symptoms.